Mallinckrodt PLC presented the first results from its two-part pilot study’s 44-week open label extension period, on the efficacy of H.P. Acthar Gel, a repository corticotropin injection, in persistent systemic lupus erythematosus (SLE).
Acthar is approved by the U.S. Food and Drug Administration for flares, or for maintenance therapy, in certain SLE patients.
The eight-week, double-blind, placebo-controlled, randomized study, “A two-part study exploring the efficacy, safety, and pharmacodynamics of Acthar in systemic lupus erythematosus patients with a history of persistently active disease” was presented at the European League Against Rheumatism Annual Congress (EULAR 2016), June 8-11 in London.
“Systemic Lupus Erythematosus is a disease that can be very difficult to manage,” said Dr. Richard A. Furie, chief of the Division of Rheumatology for Northwell Health in Greatneck New York, in a press release. “It is encouraging to see clinically meaningful, longer-term results from this pilot study of repository corticotropin injection, and I look forward to future data readouts.”
Patients who completed the first phase of the trial were able to volunteer for the second phase. All subjects in the 44-week open label phase received Acthar, including those who received placebo in the initial double-blind phase.
No unexpected adverse events occurred although five participants withdrew from the trial due to potential side effects during the extended phase.
The open label extended study evaluated the effect of RCI on SLE symptoms – including tender and swollen joints. According to the press release, despite the study’s limitations that included a small sample size and lack of blinding and comparator group, study results showed that:
- Participants with persistently active SLE who continued on RCI during the 44-week period sustained the achieved reductions in specific measures of SLE activity during the blinded phase.
- Participants who switched from placebo to Acthar period reported improvements in some measures of SLE activity. By 12 to 16 weeks after beginning RCI dosing, patients who were given placebo in the double-blind study phase reported improvements that were generally comparable to the improvements seen in patients receiving RCI treatment in the blinded, weigh-week phase of the study.
- Nearly 1/3 of participants were able to lower exogenous corticosteroid dosing by at least 50%, throughout the full study’s 52 weeks.
“Taken together, the data from the two phases of this pilot study provide insight into the potential efficacy of Acthar’s use as a treatment option in SLE patients who have persistent disease activity despite corticosteroid therapy,” said Dr. Steven Romano, Mallinckrodt’s senior vice president and chief scientific officer. “The findings support the advancement of a well-powered, confirmatory trial in this lupus population, and Mallinckrodt plans to begin this company-sponsored study later this year.”